Open Label Study of R788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)

December 6, 2023 updated by: Rigel Pharmaceuticals

A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura

The primary objective of this study was to assess the long term safety of fostamatinib in subjects with persistent/chronic ITP

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
123

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Concord, New South Wales, Australia, 2139
        • Concord Repatriation General Hospital
      • Liverpool, New South Wales, Australia, 2170
        • Liverpool Hospital
      • Randwick, New South Wales, Australia, 2031
        • Prince of Wales Hospital
      • Westmead, New South Wales, Australia, 2145
        • Westmead Hospital
    • Tasmania
      • Launceston, Tasmania, Australia, 7250
        • Launceston General Hospital
    • Victoria
      • Melbourne, Victoria, Australia, 3004
        • The Alfred
    • Western Australia
      • Nedlands, Western Australia, Australia, 6009
        • Perth Blood Institute
  • Austria
      • Vienna, Austria, 1140
        • Hanusch-Krankenhaus Wiener Gebietskrankenkasse
  • Bulgaria
      • Pleven, Bulgaria, 5800
        • UMHAT Dr. Georgi Stranski, EAD, Pleven, Clinic of Hematology
      • Sofia, Bulgaria, 1431
        • UMHAT Aleksandrovska, EAD
      • Vratsa, Bulgaria, 3000
        • MHAT Hristo Botev, AD, Vratsa, First Internal Department
    • BG
      • Sofia, BG, Bulgaria, 1756
        • Specialized Hospital for Active Treatment of Hematology Diseases, EAD, Sofia, Department of Chemotherapy, Hemotherapy and Blood Inherited Diseases to Clinic of Clinical Hematology;
  • Canada
    • Ontario
      • Hamilton, Ontario, Canada, L8N 3Z5
        • Hamilton Health Sciences Corporation
      • Toronto, Ontario, Canada, M5B1W8
        • St. Michael's Hospital
  • Czechia
      • Brno, Czechia, 625 00
        • Fakultní Nemocnice Brno
      • Ostrava-Poruba, Czechia, 708 52
        • Fakultni nemocnice Ostrava
  • Denmark
    • DK
      • Herlev, DK, Denmark, 2730
        • Herlev Hospital
  • Hungary
      • Pecs, Hungary, H-7624
        • Pecsi Tudomanyegyetem Klinikai Kozpont, I. sz. Belgyogyaszati Klinika
  • Italy
      • Udine, Italy, 33100
        • Azienda Ospedaliero-Universitaria "Santa Maria della Misericordia" - Clinica Ematologica
    • BO
      • Bologna, BO, Italy, 40138
        • Istituto di Ematologia "Lorenzo e Ariosto Seràgnoli"
  • Netherlands
    • NL
      • Den Haag, NL, Netherlands, 2545 CH
        • Haga Ziekenhuis
  • Norway
      • Bergen, Norway, 5021
        • Haukeland universitetssykehus, Helse Bergen HF
      • Grålum, Norway, 1714
        • Sykehuset Østfold Kalnes
  • Poland
      • Gdansk, Poland, 80-952
        • Lkinika Hematologii I Transplantologii Uniwersyteckie Centrum Kliniczne
      • Kraków, Poland, 31-501
        • SPZOZ Szpital Uniwersytecki w Krakowie Pracownia Separacji Krwinek i Bank Komórek Krwiotwórczych Klinika Hematologii
      • Lodz, Poland, 93-510
        • Wojewódzki Szpital Specjalistyczny im. M. Kopernika w Łodzi
      • Lublin, Poland, 20-601
        • Specjalistyczny Gabinet Lekarski
      • Opole, Poland, 45-061
        • Szpital Wojewodzki w Opolu
      • Warszawa, Poland, 02-776
        • Instytut Hematologii I Transfuzjologii
    • Dolnoslaski
      • Wroclaw, Dolnoslaski, Poland, 50-367
        • Samodzielny Publiczny Szpital Kliniczny Nr 1 we Wrocrlaw
    • PO
      • Slupsk, PO, Poland, 76-200
        • Wojewodzki Szpital Specjalistyczny im. J. Korczaka
  • Romania
      • Bucuresti, Romania, 020125
        • Spitalul Clinic Colentina, Hematologie
  • Spain
      • Barcelona, Spain, 08035
        • Hospital Universitari Vall d'Hebron
      • Madrid, Spain, 28046
        • Hospital Universitario La Paz
      • Valencia, Spain, 46026
        • Hospital Universitari i Politecnic La Fe de Valencia
  • United Kingdom
      • Canterbury, United Kingdom, CT1 3NG
        • Kent & Canterbury Hospital
      • Great Yarmouth, United Kingdom, NR31 6LA
        • James Paget University Hospital
      • Leeds, United Kingdom, LS9 7TF
        • St. James's Hospital
      • Leicester, United Kingdom, LE1 5WW
        • Leicester Royal Infirmary
      • Liverpool, United Kingdom, L78XP
        • Royal Liverpool University Hospital
      • London, United Kingdom, W12 0HS
        • Imperial College Healthcare NHS Trust
      • London, United Kingdom, WC1E 6AG
        • University College Hospital
      • Oxford, United Kingdom, OX3 7LE
        • Cancer and Haematology Centre
      • Stoke-on-Trent, United Kingdom, ST4 6QG
        • University Hospital of North Midlands NHS Trust, Royal Stoke University Hospital
    • Essex
      • Colchester, Essex, United Kingdom, CO4 5JL
        • Colchester General Hospital
    • UK
      • Newcastle-upon-Tyne, UK, United Kingdom, NE1 4LP
        • Royal Victoria Infirmary
  • United States
    • Arizona
      • Tucson, Arizona, United States, 85710
        • Arizona Oncology Associates
    • Illinois
      • Peoria, Illinois, United States, 61615
        • Bleeding & Clotting Disorders Institute
    • Indiana
      • Lafayette, Indiana, United States, 47905
        • Horizon Oncology Research, Inc
    • Maryland
      • Bethesda, Maryland, United States, 20817
        • Center for Cancer and Blood Disorders
    • New York
      • New York, New York, United States, 10065
        • Weill Cornell Medicine
      • New York, New York, United States, 10065
        • Weill Cornell Medical College/New York Presbyterian Hospital
    • North Carolina
      • Greenville, North Carolina, United States, 27834
        • East Carolina University, Brody School of Medicine
      • Salisbury, North Carolina, United States, 28144
        • W.G. "Bill" Hefner VA Medical Center
    • Ohio
      • Middletown, Ohio, United States, 45042
        • Signal Point Clinical Research Center LLC

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Completed week 24 evaluation of Study C935788-047 or Study C935788-048 or discontinued early due to lack of response.
  • Able and willing to give written informed consent

Exclusion Criteria:

  • Discontinued participation in Study C935788-047 or Study C935788-048 for any reason other than lack of response
  • Poorly controlled hypertension during Study C935788-047 or Study C935788-048
  • Significant infection, an acute infection such as influenza, or known inflammatory process

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Fostamatinib Disodium
Fostamatinib Disodium tablet 100 mg or 150 mg by mouth twice a day
Drug: Fostamatinib Disodium
Fostamatinib Disodium tablet 100 mg or 150 mg by mouth twice a day
Other Names:
  • R935788
  • Fostamatinib
  • R788

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Percentage of Subjects Who Achieved Platelet Count of at Least 50,000/µL Within 12 Weeks of Beginning Treatment up to 12 Months (Fostamatinib in 047/048 or 049):Version 1
Time Frame: Up to 12 months
Percentage of subjects who achieved platelet count of at least 50,000/µL within 12 Weeks of beginning treatment up to 12 months was analyzed among all subjects who received active treatment in one of the prior studies (C788-047 or C788-048), in the current extension study (C788-049), or in both prior and current studies. Treated Population was defined as all enrolled and treated subjects.
Up to 12 months
Percentage of Subjects Who Achieved Platelet Count of at Least 50,000/µL Within 12 Weeks of Beginning Treatment up to 12 Months (Placebo in 047/048 and Fostamatinib 049): Version 2
Time Frame: Up to 12 months
A within-subject, between-study comparison of platelet counts for subjects who were previously treated with placebo in one of the prior studies (C788-047 or C788-048) was prospectively defined in the protocol (version 2). Achievement of platelet response by 12 weeks and maintenance of platelet response for 12 weeks was analyzed among subjects who had been randomized to placebo in one of the prior studies (C788-047 or C788-048). Treated Population was defined as all enrolled and treated subjects.
Up to 12 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Duration of Platelet Response Based on Platelet Count and Rescue Medication
Time Frame: Up to 12 months
The duration of platelet response was defined as the time from when the subject first achieved a platelet count of at least 50,000/µL, until the first of 2 visits with platelet counts < 50,000/µL that were at least 4 weeks apart without an intervening visit with a platelet count less than equal to (<=) 50,000/µL unrelated to rescue therapy. Duration of platelet response was analyzed using the Kaplan-Meier method. Treated Population was defined as all enrolled and treated subjects. Here, a number of subjects analyzed included all subjects evaluable for this endpoint.
Up to 12 months
Percentage of Subjects in Whom a Reduction in the Dose of Concomitant ITP Therapy Can be Achieved While Maintaining an Adequate Platelet Count
Time Frame: Up to 12 months
The percentage of subjects in whom a reduction in the dose of concomitant ITP therapy could be achieved while maintaining an adequate platelet count, the reduction event was clarified to apply only to reductions in the dose of concomitant ITP therapy occurring within a period of platelet response and the reduction event was not be prompted by an adverse event.
Up to 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Rigel Pharmaceuticals

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Rigel Pharmaceuticals, Inc., Rigel Pharmaceuticals, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 1, 2014

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 2, 2020

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 2, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 27, 2014

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 28, 2014

First Posted (Estimated)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 4, 2014

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
December 11, 2023

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 6, 2023

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
December 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • C-935788-049
  • 2013-005454-30 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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